Advancing Gene Therapy: Personalized Gene Editing
From BREAKING THROUGH with Madeline Bell by Children’s Hospital of Philadelphia
October 13, 2025 · 22 min · Episode 23
About this episode
This episode discusses a groundbreaking personalized gene editing therapy for a baby diagnosed with a rare metabolic disorder.
When baby KJ was diagnosed with a rare metabolic disorder, the team at Children’s Hospital of Philadelphia (CHOP) had a unique treatment option for him: a personalized gene editing therapy developed in collaboration with Penn Medicine. On this episode, Madeline speaks to CHOP geneticist Dr. Rebecca Ahrens-Nicklas, who describes how KJ’s first-of-its-kind treatment was developed – and what this amazing breakthrough means for other patients.
People in this episode
Host: Madeline Bell
Guest: Dr. Rebecca Ahrens-Nicklas
Topics covered
- gene therapy
- personalized medicine
- metabolic disorders
- genetic research
- patient care
Keywords
- gene therapy
- personalized gene editing
- metabolic disorder
- Children’s Hospital of Philadelphia
- Penn Medicine
Mentioned in this episode
Organizations: Children’s Hospital of Philadelphia, Penn Medicine
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- New Frontiers in Injury Prevention · August 15, 2024 · 26 min
- Gene Therapy for Sickle Cell Disease · June 17, 2024 · 29 min
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